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CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. Frangoul H, et al. Among authors: de montalembert m, de la fuente j. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5. N Engl J Med. 2021. PMID: 33283989 Clinical Trial.
Pregnancy in sickle cell disease is at very high risk.
de Montalembert M, Deneux-Tharaux C. de Montalembert M, et al. Blood. 2015 May 21;125(21):3216-7. doi: 10.1182/blood-2015-04-638288. Blood. 2015. PMID: 25999441 Free article. No abstract available.
Sickle cell disease.
Ware RE, de Montalembert M, Tshilolo L, Abboud MR. Ware RE, et al. Among authors: de montalembert m. Lancet. 2017 Jul 15;390(10091):311-323. doi: 10.1016/S0140-6736(17)30193-9. Epub 2017 Feb 1. Lancet. 2017. PMID: 28159390 Review.
Gene Therapy in a Patient with Sickle Cell Disease.
Ribeil JA, Hacein-Bey-Abina S, Payen E, Magnani A, Semeraro M, Magrin E, Caccavelli L, Neven B, Bourget P, El Nemer W, Bartolucci P, Weber L, Puy H, Meritet JF, Grevent D, Beuzard Y, Chrétien S, Lefebvre T, Ross RW, Negre O, Veres G, Sandler L, Soni S, de Montalembert M, Blanche S, Leboulch P, Cavazzana M. Ribeil JA, et al. Among authors: de montalembert m. N Engl J Med. 2017 Mar 2;376(9):848-855. doi: 10.1056/NEJMoa1609677. N Engl J Med. 2017. PMID: 28249145 Free article.
Recommendations regarding splenectomy in hereditary hemolytic anemias.
Iolascon A, Andolfo I, Barcellini W, Corcione F, Garçon L, De Franceschi L, Pignata C, Graziadei G, Pospisilova D, Rees DC, de Montalembert M, Rivella S, Gambale A, Russo R, Ribeiro L, Vives-Corrons J, Martinez PA, Kattamis A, Gulbis B, Cappellini MD, Roberts I, Tamary H; Working Study Group on Red Cells and Iron of the EHA. Iolascon A, et al. Among authors: de montalembert m, de franceschi l. Haematologica. 2017 Aug;102(8):1304-1313. doi: 10.3324/haematol.2016.161166. Epub 2017 May 26. Haematologica. 2017. PMID: 28550188 Free PMC article.
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.
Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarré C, Beuzard Y, Chrétien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M. Thompson AA, et al. Among authors: de montalembert m. N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342. N Engl J Med. 2018. PMID: 29669226 Free article. Clinical Trial.
Prognostic factors of disease severity in infants with sickle cell anemia: A comprehensive longitudinal cohort study.
Brousse V, El Hoss S, Bouazza N, Arnaud C, Bernaudin F, Pellegrino B, Guitton C, Odièvre-Montanié MH, Mames D, Brouzes C, Picard V, Nguyen-Khoa T, Pereira C, Lapouméroulie C, Pissard S, Gardner K, Menzel S, Le Van Kim C, Colin-Aronovicz Y, Buffet P, Mohandas N, Elie C, Maier-Redelsperger M, El Nemer W, de Montalembert M. Brousse V, et al. Among authors: de montalembert m. Am J Hematol. 2018 Nov;93(11):1411-1419. doi: 10.1002/ajh.25260. Epub 2018 Sep 21. Am J Hematol. 2018. PMID: 30132969 Free article. Clinical Trial.
A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.
Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J; HOPE Trial Investigators. Vichinsky E, et al. N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14. N Engl J Med. 2019. PMID: 31199090 Clinical Trial.
EHA Research Roadmap on Hemoglobinopathies and Thalassemia: An Update.
Iolascon A, De Franceschi L, Muckenthaler M, Taher A, Rees D, de Montalembert M, Rivella S, Eleftheriou A, Cappellini MD. Iolascon A, et al. Among authors: de montalembert m, de franceschi l. Hemasphere. 2019 Jun 4;3(3):e208. doi: 10.1097/HS9.0000000000000208. eCollection 2019 Jun. Hemasphere. 2019. PMID: 31723835 Free PMC article. Review.
225 results